| Project/Area Number |
26461552
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Jikei University School of Medicine |
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Principal Investigator |
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| Research Collaborator |
TAHARA Mayu 東京慈恵会医科大学, 医学部, 大学院生
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| Project Period (FY) |
2014-04-01 – 2018-03-31
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| Project Status |
Completed (Fiscal Year 2017)
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| Budget Amount *help |
¥4,940,000 (Direct Cost: ¥3,800,000、Indirect Cost: ¥1,140,000)
Fiscal Year 2016: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2015: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2014: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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| Keywords | てんかん / 病態 / 細胞治療 / iPS細胞 / モデルラット / MRI / 遺伝子 / 創薬 |
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| Outline of Final Research Achievements |
Dravet syndrome (DS) is an infantile-onset intractable epilepsy, mainly caused by mutation in SCN1A gene. This research aims to elucidate novel pathomechanisms on DS and to achieve a transplantation therapy of GABAergic inhibitory precursors in the future. In the present study, 1) we developed a method to efficiently induce GABAergic neurons from human induced pluripotent stem cells (iPSCs). 2) We established mutation-repaired iPSCs from a patient-derived iPSCs by using a TALEN-based genome-editing technology. 3) Finally, to identify brain regions that is involved in the epilepsy pathogenesis in DS, we employed a manganese-enhanced magnetic resonance imaging technique, which may detect regions with increased neuronal activity. We identified several enhanced regions including hippocampus, thalamus, and some neocortical areas. Although further evaluations are necessary, this method will elucidate anatomical basis for epileptogenesis and target regions for cell therapy.
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